Slide 1: 2014 Q1 Announcement
Acucela is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of individuals worldwide. 2014 Q1 Announcement Tokyo, JAPAN May 20-21, 2014
Slide 2: Disclaimer
DISCLAIMER This presentation contains forward-looking statements concerning our approved products and product development, our technology, our competitors, our intellectual property, our financial condition and our plans for research and development programs that involve risks, uncertainties and ass
Slide 3: Introduction
a ACUC ELA Introduction
Slide 4: Acucela's Vision and Mission
Acucela’s Vision and Mission Vision: Innovative ophthalmology company focused on developing therapies to treat sight- threatening diseases Mission: • To address unmet medical needs in sight-threatening diseases through scientific innovations • To build a unique company with continuous value creation
Slide 5: 2014 Q1 and Recent Highlights
2014 Q1 and Recent Highlights • February: Completed IPO on Mothers Board of Tokyo Stock Exchange (4589) • March: Completed enrollment in Phase 2b/3 “SEATTLE” study ahead of schedule; no financial impact to 2014 estimates • May: Held formal meeting with the FDA to discuss ongoing Phase 2b/3 “SEATTLE”
Slide 6: Pipeline Chart
Pipeline Chart 2014 Q1 Results 6 Drug PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 Product Origin (2) Acucela Territory IP Expiration Date Emixustat HCI Acucela North America (joint), EU, South America and Africa, Nov 2029 (Subject to extension)(3) Other VCM and retina product candidates Global OPA-6566 Ots
Slide 7: Emixustat Hydrochloride
Pat ACUCELA Emixustat Hydrochloride
Slide 8: Drug Development Path
Drug Development Path Emixustat for GA associated with dry AMD: Investigational compound based on Acucela’s proprietary VCM 2014 Q1 Results 8 2005 2007 2009 2012 2013 » Phase 2b/3 trial commences » Two-year treatment » Toxicology » Proof-of-concept in preclinical model » Medical chemistry » Five Pha
Slide 9: Phase 2b/3 Emixustat Clinical Trial
Phase 2b/3 Emixustat Clinical Trial 2014 Q1 Results 9 • Design − Randomized, double-masked, dose-ranging study comparing the safety and efficacy of emixustat with placebo in patients with GA associated with dry AMD • Objectives − Primary objective – Determine if emixustat reduces the rate of GA lesi
Slide 10: Summary of the May 9th Press Release
Summary of the May 9th Press Release 2014 Q1 Results 10 Recent update on Emixustat Hydrochloride Clinical Development Program • Key Takeaways and Next Steps - Emixustat Hydrochloride Phase 2b/3 “SEATTLE” Study in Patients with GA Associated with Dry AMD − Acucela intends to continue the study throug
Slide 11: Financial Overview
a ACUC ELA Financial Overview
Slide 12: Financial Arrangements with Otsuka Pharmaceutical: Emixustat Agreement
Financial Arrangements with Otsuka Pharmaceutical • Key points on financial arrangements − Emixustat Agreement is a co-development and collaboration agreement to develop and commercialize emixustat and/or other back-up compounds for the treatment of dry AMD and other potential ophthalmic indications
Slide 13: Financial Arrangements with Otsuka Pharmaceutical: Glaucoma Agreement
Financial Arrangements with Otsuka Pharmaceutical • Key points on financial arrangements − The Glaucoma Agreement provides us the right to exercise our option (“opt-in right”) to co-develop and co-promote OPA-6566 in the US upon the success of Phase 2 and/or Phase 3 clinical trials − Otsuka will fun
Slide 14: P/L: 2013 Q1 and 2014 Q1
P/L: 2013 Q1 and 2014 Q1 15,980 10,546 5,720 215 3,711 54 - 2,000 4,000 6,000 8,000 10,000 12,000 14,000 16,000 18,000 2013Q1 2014Q1 Revenue from collaborations Income from operations Net income 2014 Q1 Results 14 (In thousand US dollars) Note: Unaudited results Source: Acucela Inc. Form 10-Q • Reve
Slide 15: B/S: 12/31/2013 and 3/31/2014
B/S: 12/31/2013 and 3/31/2014 11,767 51,054 42,281 142,330 31,124 185,317 - 50,000 100,000 150,000 200,000 250,000 Dec-31-13 Mar-31-14 Dec-31-13 Mar-31-14 Total shareholders' equity Current assets Long-term assets 2014 Q1 Results 15 (In thousand US dollars) Note: Unaudited results Source: Acucela In
Slide 16: Financial Overview: P/L
Financial Overview: P/L 27,019 36,457 34,226 46,424 52,947 61,840 2,350 4,528 3,869 7,033 6,994 836 2,113 9,003 6,266 4,178 4,299 264 - 10,000 20,000 30,000 40,000 50,000 60,000 70,000 FY2009 FY2010 FY2011 FY2012 FY2013 2014 est Revenue from collaborations Income from operations Net income 2014 Q1 R
Slide 17: Financial Overview: B/S
Financial Overview: B/S 2014 Q1 Results 17 23,638 34,809 41,495 47,024 54,048 193,384 4,209 14,101 20,840 25,607 31,124 185,317 - 50,000 100,000 150,000 200,000 250,000 FY2009 FY2010 FY2011 FY2012 FY2013 2014 Q1 Total assets Total shareholders' equity (In thousand US dollars) Note: 2009 and 2010 and
Slide 18: Acucela is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics
Acucela is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of individuals worldwide.